Clinical trials: Demyelinating Diseases
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Multiple Sclerosis
Endeavor™ in Pediatric MS
This study will examine the feasibility of using an Endeavor™ application as a treatment modality for cognitive impairments in the pediatric MS population.
- Trial name
- Akili (NCT04445116)
- Testing
- Action Video Game Treatment
- Where
- United States
- Sponsor
- NYU Langone Health
Neurologic Signs and Symptoms
Incidence, and Survival Medical Expenditure of Patients With Multiple Sclerosis
Multiple sclerosis (MS) is a disease of autoimmune system which attacks the central nervous system and that is one of the most common inflammatory diseases and has caused a heavy disease burden on patients.
- Trial name
- NCT04022564
- Where
- Taiwan
- Volunteers
- 2,000 people
- Sponsor
- Chun-Ming Liao
Multiple Sclerosis
MR Perfusion Imaging and Hypercapnia (Increased Carbon Dioxide) to Study New Blood Vessel Formation in Multiple Sclerosis
This study will use magnetic resonance imaging (MRI) to examine and compare changes in blood flow and blood volume in the brains of normal volunteers and patients with multiple sclerosis (MS).
- Trial name
- NCT00064909
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Neuromyelitis Optica Spectrum Disorders (NMOSD)
HANDLE-a Real World Study on Satralizumab in NMOSD
This study is a single-center, retrospective-prospective, non-interventional cohort study to assess the clinical outcomes of Chinese NMOSD patients treated with satralizumab in a real-world patient management model by collecting…
- Trial name
- NCT06829524
- Testing
- Satralizumab
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Huashan Hospital
Relapsing-remitting Multiple Sclerosis
A Study to Evaluate Safety and Efficacy of Ocrelizumab in Comparison With Fingolimod in Children and Adolescents With Relapsing-remitting Multiple Sclerosis (RRMS)
This double-blind, double-dummy study will evaluate the safety and efficacy of ocrelizumab compared with fingolimod in children and adolescents with RRMS aged between 10 and < 18 years over a flexible duration.
- Trial name
- Operetta 2 (NCT05123703)
- Testing
- Ocrelizumab, Fingolimod
- Where
- 71 sites in United States, Argentina and 23 more
- Volunteers
- 188 people
- Sponsor
- Hoffmann-La Roche
Krabbe Disease
Krabbe Disease Global Patient Registry
The purpose of this study is to develop a clinical database of individuals diagnosed with Krabbe disease in order to determine which symptoms herald the onset of clinical disease in the various phenotypes of Krabbe disease; to…
- Trial name
- NCT02993796
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- State University of New York at Buffalo
Neuromyelitis Optica Spectrum Disorder
Clinical Patterns of Neuromyelitis Optica Spectrum Disorders in Assiut University Hospital
Neuromyelitis Optica Spectrum Disorders (NMOSD) are a group of inflammatory demyelinating disorders of the central nervous system.
- Trial name
- NCT03819413
- Testing
- serum aquaporin 4 antibody (AQP-4-Ab), serum MOG antibody (anti-MOG), MRI brain, spine and orbit
- Where
- Egypt
- Volunteers
- 90 people
- Sponsor
- Assiut University
Secondary Progressive Multiple Sclerosis (SPMS)
Long Term Special Drug Use-results Surveillance for Mayzent in SPMS Patients
This study is a single cohort, central registration system, all-case, open-label, multicenter observational study in patients using Mayzent for the indication of secondary progressive multiple sclerosis.
- Trial name
- NCT04593927
- Testing
- Mayzent
- Where
- 174 sites in Japan
- Volunteers
- 451 people
- Sponsor
- Novartis Pharmaceuticals
Meningitis, Encephalitis
The Effectiveness and Safety of Human Lumbar Puncture Assist Device (LPat)
The purpose of this study is to proof and investigate the effectiveness and safety of the invented device named "Human Lumbar Puncture Assist Device (LPat)" as an assist tool to be utilized to improve the success rate of…
- Conditions
- Meningitis, Encephalitis, Guillain-Barré, Subarachnoid Hemorrhage
- Trial name
- LPat (NCT03710278)
- Testing
- LPat Device
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Spartanburg Regional Healthcare System
Metachromatic Leukodystrophy (MLD)
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
- Trial name
- NCT00418561
- Testing
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Multiple Sclerosis
Safety and Efficacy in Pediatric MS Patients Prescribed Tysabri
The primary objective of the study is to evaluate the safety of natalizumab use in the pediatric multiple sclerosis (MS) population.
- Trial name
- NCT02137109
- Testing
- natalizumab
- Volunteers
- 400 people
- Sponsor
- Biogen
Multiple Sclerosis
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon [IFN]-beta-1a) in children and adolescents, using information already recorded in medical records.
- Trial name
- REPLAY (NCT01207648)
- Testing
- Rebif®
- Where
- 18 sites in United States, Argentina and 6 more
- Volunteers
- 307 people
- Sponsor
- EMD Serono
Multiple Sclerosis
A Study on the Safety of Ozanimod Exposure in Pregnant Women and Their Offspring
The purpose of this study is to provide information on maternal, fetal, and infant outcomes following exposure of ozanimod during pregnancy so that participants and physicians can weigh the benefits and risks of exposure to the…
- Trial name
- NCT05170126
- Where
- 3 sites in United States, Germany
- Volunteers
- 1,182 people
- Sponsor
- Bristol-Myers Squibb
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Multiple Sclerosis, Ataxia
"Evaluation of Usability and Safety of the Self-balancing Walking System Atalante in Patients With Multiple Sclerosis"
Multiple sclerosis (MS) is the most prevalent chronic inflammatory disease of the central nervous system (CNS), affecting more than 2 million people worldwide,1 it is a degenerative disease that selectively affects the central…
- Conditions
- Multiple Sclerosis, Ataxia, Paraplegia
- Trial name
- EXO (NCT05563402)
- Testing
- Atalante
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- Multiple Sclerosis Center of Catalonia
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Vaccine-induced Thrombotic Thrombocytopenia Syndrome, Guillain-Barre Syndrome
Gene Sequencing as a Strategy for Identifying Genetic Factors Associated With Serious Adverse Events After Covid-19 Vaccines in Use in Brazil
This protocol proposes to investigate genetic factors that may be involved in the pathogenesis of adverse events of interest with selected covid-19 vaccines: vaccine-induced immune thrombotic thrombocytopenia, and neurological…
- Conditions
- Vaccine-induced Thrombotic Thrombocytopenia Syndrome, Guillain-Barre Syndrome, Acute Disseminated Encephalomyelitis, Transverse Myelitis
- Trial name
- NCT05630313
- Where
- Brazil
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- The Immunobiological Technology Institute (Bio-Manguinhos) / Oswaldo Cruz Foundation (Fiocruz)